The European Medicines Agency (EMA) has backed a digital ambulation measure using wearables developed by Sysnav Healthcare as a primary endpoint in trials involving Duchenne muscular dystrophy (DMD) patients aged four years and older
Stride Velocity 95th centile (SV95C) measures peak ambulatory performance of patients captured during normal daily living and, according to Sysnav, provides “an accurate and clinically meaningful insight into real-world functional ability of patients and measures the progression of their disease or efficacy of therapies”.
The EMA’s decision, which qualifies the measure as an alternative to the widely used six-minute walking test, makes SV95C the first digital endpoint to be endorsed by a regulatory agency as a primary endpoint in pivotal studies.
DMD is a rare and progressive neuromuscular genetic disease that occurs in approximately one in every 3,500 to 5,000 newborn males worldwide.
The condition is caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.
Over time, this causes progressive loss of muscle strength, with most patients requiring full-time use of a wheelchair by their early teens. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure.
While there has been significant promise in new therapy development for DMD, Sysnav explained that measuring the impact of an intervention can be difficult due to the heterogeneity of disease progression.
SV95C, which can be captured to a regulatory standard by Sysnav’s ActiMyo and Syde wearable devices, could address many of the limitations of traditional endpoints currently used in clinical DMD trials.
Damien Eggenspieler, Sysnav Healthcare programme director, said: “We truly believe in the transformative impact of digital endpoints in clinical development – enabling better measurement of the functional ability of DMD patients and to assess potential treatment benefits more objectively and reliably.
“The digital endpoint SV95C has the potential to shorten clinical trial duration or decrease the number of patients.”
The EMA backing is the result of a 14-year collaboration between stakeholders across the DMD community, Sysnav said, including technology developers, academia, the pharmaceutical industry, caregivers and patients and families living with DMD.